Drug · Small Molecule

Everolimus

CI-DRUG-00000226Explore in graph →NCIt C48387 CHEMBL1908360 CIViC Approved

Regulatory

Approvals (31)

Each record names the authority, jurisdiction, indication text and status. A drug approved in one jurisdiction for one indication is not 'approved' in general.

openfdaRegulatory29 approval records · authority, jurisdiction and indication text as published.

Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
US FDA	—	Efficacy supplement 2021-12-30 (see label)	approved	Dec 30, 2021	openfda
US FDA	—	Efficacy supplement 2018-04-10 (see label)	approved	Apr 10, 2018

Data updated 17 hours agoSource updated unknown

Derived

Development pipeline

Most advanced stage across all cancers, then per top-level cancer reached through trial conditions or approval indications. Approval in any ingested jurisdiction outranks trial phase; counts are interventional studies.

Scope	Stage	Max phase	Active	Recruiting	Phase 3	Trials	Approvals	Jurisdictions	First approval	First trial
All cancers	Approved	Phase 4	70	29	54	509	31	EU, US	Mar 30, 2009	Nov 13, 2002
Malignant Breast Neoplasm	Approved	Phase 3	16	8	14	76	2	US	Mar 30, 2009	May 10, 2005
Malignant Kidney Neoplasm	Approved	Phase 4	5	0	7	62	3	EU, US	Mar 30, 2009	2004-12
Malignant Liver Neoplasm	Phase4	Phase 4	2	0	0	13	0

Curated evidence

Clinical evidence (44)

CIViC items in which this therapy appears, grouped by cancer context, then molecular profile. 50 items per page.

civicCuratedShowing 1–44 of 44 evidence items · levels, directions and significance as curated at the source; each row links to its CIViC record.

Therapy	Cancer	Type	Level	Direction · significance	Rating (1–5)	Status	Evidence	Source
MTOR Mutation1
Everolimus + Pazopanib

Clinical trials

Trials with this intervention (518)

Most recently updated first, 50 per page.

clinicaltrialsPublishedShowing 301–350 of 518 studies · status and phase as posted by the registrant; each row links to the study record.

NCT	Title	Status	Phase	Enrollment (n)	Sponsor	Countries	Last update	Source
NCT01031446	Cisplatin, Paclitaxel, and Everolimus in Treating Patients With Metastatic Breast Cancer	completed	Phase 1 / Phase 2	55	Vanderbilt-Ingram Cancer CenterOTHER	1	Aug 31, 2018	clinicaltrials
NCT00954512	Study of Robatumumab (SCH 717454, MK-7454) in Combination With Different Treatment Regimens in Participants With Advanced Solid Tumors (P04722, MK-7454-004)	terminated

Molecule

Record

Kind: Small Molecule
Mechanism: FK506-binding protein 1A inhibitor
Targets: 1 gene
UNII: 9HW64Q8G6G
Aliases: (1R,9S,12S,15R,16E,18R,19R,21R,23S,24E,26E,28E,30S,32S,35R)-1,18-Dihydroxy-12-((1R)-2-((1S,3R,4R)-4-(2-hydroxyethoxy)-3-methoxycyclohexyl)-1-methylethyl)-19,30-dimethoxy-15,17,21,23,29,35-hexamethyl-11,36-dioxa-4-azatricyclo(30.3.1.04,9)hexatriaconta-16,24,26,28-tetraene-2,3,10,14,20-pentaone, 40-o-(2-hydroxyethyl)-rapamycin, 42-o-(2-hydroxyethyl)rapamycin, 42-O-(2-Hydroxy)ethyl Rapamycin, Afinitor, AFINITOR, AFINITOR DISPERZ, Certican, Everolimus, RAD 001, RAD-001, RAD001, RAD 666, Rapamycin, 42-o-(2-hydroxyethyl)-, Sdz rad, SDZ-RAD, TORPENZ, Votubia, YULITHIRA, Zortress, ZORTRESS

Data updated 20 hours agoSource updated unknown

Identifiers

Codes (3)

Upstream identifiers kept as first-class codes; each links to its registry where a stable public URL exists.

System	Code	Label
ATC (WHO)	L01XE10	Antineoplastic agents
EMA product	EMEA/H/C/001038

PTEN Loss4
Everolimus	Bladder Carcinoma	Predictive	B	Supports Resistance	4	accepted	EID644 PTEN loss is associated with reduced response to mTOR inhibitors in patients with bladder cancer. PMID 23989949 · Seront et al., 2013 · Open in CIViC	civic
〃	HER2-Positive Breast CarcinomaALIAS	Predictive	B	Supports Sensitivity Response	4	accepted	EID1297 Archival tumor samples from 549 patients from the BOLERO-1 and -3 trials were analyzed for alterations in the PI3K pathway using next-generation sequencing, immunohistochemistry (IHC) and Sanger seque… (full text at CIViC) PMID 27091708 · André et al., 2016 · Open in CIViC	civic
Everolimus + MTOR Kinase Inhibitor PP242Substitutes	Malignant Breast Neoplasm	Predictive	D	Does Not Support Sensitivity Response	3	accepted	EID1611 In a panel of 31 breast cancer cell lines, 11 had loss of PTEN function. Cells were tested for sensitivity to mTORC1 inhibitor everolimus and mTORC1/2 inhibitor PP242, and no significant correlation w… (full text at CIViC) PMID 21358673 · Weigelt et al., 2011 · Open in CIViC	civic
Everolimus	Malignant NeoplasmALIAS	Predictive	B	Does Not Support Sensitivity Response	3	accepted	EID5840 In a single-arm, open label phase 2 trial in PIK3CA amplified/mutated and/or PTEN loss encompassing 10 patients, no objective responses were observed but a stable disease response in 4 patients, resul… (full text at CIViC) PMID 28330462 · Kim et al., 2017 · Open in CIViC	civic

TSC1 Loss-of-function2
Everolimus	Bladder Carcinoma	Predictive	B	Supports Sensitivity Response	3	accepted	EID155 Tumors (n=14) expressing TSC1 mutations (5/14) show notably more sensitivity to everolimus than their wild-type counterparts (9/14). A dramatic response was observed in a patient with combination of L… (full text at CIViC) PMID 22923433 · Iyer et al., 2012 · Open in CIViC	civic
〃	Tuberous SclerosisUNRESOLVED	Predictive	A	Supports Sensitivity Response	5	submitted	EID7280 In phase 3 trial patients with subependymal giant cell astrocytomas related to tuberous sclerosis complex (TSC) were randomly assigned to everolimus or placebo. Tumor response was observed 35% in ever… (full text at CIViC) PMID 23158522 · Franz et al., 2013 · Open in CIViC	civic

BRAF Fusion2
Everolimus + TrametinibCombination	Childhood Low Grade Glioma	Predictive	D	Supports Sensitivity Response	3	rejected	EID7197 This preclinical study proposed to use a combination therapy of an MEK inhibitor (e.g. trametinib) and an mTOR inhibitor (e.g. everolimus) for the treatment of pediatric low-grade gliomas (PLGGs) with… (full text at CIViC) PMID 29156677 · Jain et al., 2017 · Open in CIViC	civic
〃	Childhood Low Grade Glioma	Predictive	D	Supports Sensitivity Response	2	accepted	EID7199 This preclinical study proposed to use a combination therapy of an MEK inhibitor (e.g. trametinib) and an mTOR inhibitor (e.g. everolimus) for the treatment of pediatric low-grade gliomas (PLGGs) with… (full text at CIViC) PMID 29156677 · Jain et al., 2017 · Open in CIViC	civic

PIK3CA Mutation2
Everolimus	HER2-Positive Breast CarcinomaALIAS	Predictive	B	Supports Sensitivity Response	4	accepted	EID1296 Archival tumor samples from 549 patients from the BOLERO-1 and -3 trials were analyzed for alterations in the PI3K pathway using next-generation sequencing, immunohistochemistry (IHC) and Sanger seque… (full text at CIViC) PMID 27091708 · André et al., 2016 · Open in CIViC	civic
Everolimus + MTOR Kinase Inhibitor PP242Substitutes	Malignant Breast Neoplasm	Predictive	D	Supports Sensitivity Response	3	accepted	EID1610 A panel of 31 breast cancer cell lines was tested for sensitivity to mTORC1 inhibitor everolimus and mTORC1/mTORC2 inhibitor PP242. Inhibitor concentrations for 50% survival fraction (SF50) were measu… (full text at CIViC) PMID 21358673 · Weigelt et al., 2011 · Open in CIViC	civic

MTOR E1799K2
Everolimus	Focal Cortical DysplasiaUNRESOLVED	Predictive	D	Supports Sensitivity Response	4	submitted	EID9489 The L2427P (c.7280T>C) variant of mTOR was found in brain tissue samples from 2 focal cortical dysplasia type II (FCDII) patients at allelic frequencies of 9.6–12.6% and 6.9–7.3% in biological replica… (full text at CIViC) PMID 25799227 · Lim et al., 2015 · Open in CIViC	civic
〃	MegalencephalyUNRESOLVED	Predictive	D	Supports Sensitivity Response	3	submitted	EID9486 Three siblings had a phenotype which overlaps with known megalencephaly syndromes and RASopathies, including Costello syndrome, and were all heterozygous for mTOR c.5395G>A; p.Glu1799Lys. Peripheral b… (full text at CIViC) PMID 25851998 · Baynam et al., 2015 · Open in CIViC	civic